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| Name | Class |
|---|---|
| University Children's Hospital Basel | OTHER |
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The study aims to investigate immunomodulatory effects of thrombopoietin-receptor Agonist (TPO-RA) in patients with primary ITP, who failed first-line therapy or who became intolerant to it. It is hypothesized that the early phase of this autoimmune disease may exhibit a stronger immunomodulatory potential in response to a stimulus, such as romiplostim. Such a process may subsequently be capable to induce regulatory mechanisms or tolerance.
Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Romiplostim | Experimental | Romiplostim (a thrombopoietin-receptor agonist, TPO-RA) will be administered subcutaneously once weekly over 22 weeks with a starting dose of 1mcg/kg body weight. The dose will be adjusted based on platelet counts as described in the summary of Product Characteristics (SmPC). Followup examination at week 52. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| romiplostim | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Interleukin (IL)-4 concentrations (pg/ml) from baseline to week 22 | The primary aim of the study is to demonstrate an immunomodulatory effect of the study drug. Investigators expect a shift in the Th1/Th2 balance towards Th2. The primary outcome is to compare the pre- and post-treatment IL-4 concentrations (pg/ml) of all included patients (Th2 profile). Assessment of change in pre- and post-treatment IL-4 concentrations (pg/ml). | baseline and 22 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Change in immunomodulation as assessed by immune cell characteristics between baseline and week 22 | Immunologic parameters will be investigated between baseline and week 22 of the study: immune cell characteristics fluorescence-activated cell sorting (FACS): B cells: cluster of Differentiation Antigen (CD)3- cluster of Differentiation Antigen (CD)19+ Memory B lymphocytes CD19+cluster of Differentiation Antigen (CD)10-cluster of Differentiation Antigen (CD)27+cluster of Differentiation Antigen (CD)38- Plasma cells: CD19+ CD10- cluster of Differentiation Antigen (CD)20- CD27++ cluster of Differentiation Antigen (CD)38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ cluster of Differentiation Antigen (CD)8+ natural killer (NK) cells CD3- cluster of Differentiation Antigen (CD)16+ cluster of Differentiation (CD)56+ Monocytes CD56- cluster of Differentiation Antigen (CD)14 (low) CD16+ cluster of Differentiation Antigen (CD)33 Tregs CD4+ cluster of Differentiation Antigen (CD)25 superhigh FoxP3+ |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Thomas Kühne, Prof.Dr.med | UKBB | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Liestal Cantonal Hospital | Liestal | Basel-Landschaft | Switzerland | |||
| Lucerne Cantonal Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37735543 | Derived | Schifferli A, Rufer A, Rovo A, Nimmerjahn F, Cantoni N, Holbro A, Favre G, Dirks J, Wieland A, Faeth H, Pereira R, Kuhne T. Immunomodulation with romiplostim as a second-line strategy in primary immune thrombocytopenia: The iROM study. Br J Haematol. 2023 Oct;203(1):119-130. doi: 10.1111/bjh.19074. |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C488777 | romiplostim |
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| baseline and 22 weeks |
| Change in immunomodulation as assessed by immune cell characteristics between baseline and week 10 | Immunologic parameters will be investigated between baseline and week 10 of the study: immune cell characteristics FACS: B cells: CD3- CD19+ Memory B lymphocytes CD19+CD10-CD27+CD38- Plasma cells: CD19+ CD10- CD20- CD27++ CD38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ CD8+ NK cells CD3- CD16+ CD56+ Monocytes CD56- CD14 (low) CD16+ CD33 Tregs CD4+ CD25 superhigh FoxP3+ | baseline and 10 weeks |
| Change in immunomodulation as assessed by messenger ribonucleic acid (mRNA) of cytokines between baseline and week 22 | mRNA of cytokines will be investigated between baseline and week 22 mRNA essays (real-time quantitative PCR): cytokine mRNA (interleucin (IL)2, interleucin (IL)4, interleucin (IL)6, interleucin (IL)10, IL17, interleucin (IL)35, IFNgamma, TNFalpha, transforming growth factor (TGF)-β) | baseline and 22 weeks |
| Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 22 | mRNA of immune cells will be investigated between baseline and week 22 mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3) | baseline and 22 weeks |
| Change in immunomodulation as assessed by mRNA of cytokines between baseline and week 10 | mRNA of cytokines will be investigated between baseline and week 10 mRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β) | baseline and 10 weeks |
| Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 10 | mRNA of immune cells will be investigated between baseline and week 10 mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3) | baseline and 10 weeks |
| Change in immunomodulation as assessed by cytokine concentrations between baseline and week 22 | cytokine concentration will be investigated between baseline and week 22 ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β | baseline and 22 weeks |
| Change in immunomodulation as assessed by cytokine concentrations between baseline and week 10 | cytokine concentration will be investigated between baseline and week 10 ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β | baseline and 10 weeks |
| Clinical response between baseline and week 52: number of severe bleeding | Clinical characterization of response to romiplostim therapy will be assessed additionally : measurement of severe bleeding (score Bolton-Maggs) | baseline and 52 weeks |
| Clinical response between baseline and week 52: number of days in hospital | Clinical characterization of response to romiplostim therapy will be assessed additionally : need of inpatient daycare | baseline and 52 weeks |
| Clinical response between baseline and week 52: platelet more than >100G/l | Clinical characterization of response to romiplostim therapy will be assessed additionally : assessment of platelet response to romiplostim, according to the definitions of Rodeghiero et al. (49). | baseline and 52 weeks |
| Change in immunomodulation as assessed by immune cell characteristics between baseline and week 52 | Immunologic parameters will be investigated between baseline and week 52 of the study: immune cell characteristics FACS: B cells: CD3- CD19+ Memory B lymphocytes CD19+CD10-CD27+CD38- Plasma cells: CD19+ CD10- CD20- CD27++ CD38++ Tcells and subpopulations: CD3+/ CD3+ CD4+/ CD3+ CD8+ NK cells CD3- CD16+ CD56+ Monocytes CD56- CD14 (low) CD16+ CD33 Tregs CD4+ CD25 superhigh FoxP3+ | baseline and 52 weeks |
| Change in immunomodulation as assessed by mRNA of immune cells between baseline and week 52 | mRNA of immune cells will be investigated between baseline and week 52 mRNA essays (real-time quantitative PCR): mRNA Th1 (T-bet), Th2 (GATA-3), Th17 (RORγt), Tregs (Foxp3) | baseline and 52 weeks |
| Change of immunomodulation as assessed by mRNA of cytokines between baseline and week 52 | mRNA of cytokines will be investigated between baseline and week 52 mRNA essays (real-time quantitative PCR): cytokine mRNA (IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF-β) | baseline and 52 weeks |
| Change in immunomodulation as assessed by cytokine concentrations between baseline and week 52 | cytokine concentration will be investigated between baseline and week 52 ELISA Cytokines: IL2, IL4, IL6, IL10, IL17, IL35, IFNgamma, TNFalpha, TGF- β | baseline and 52 weeks |
| Clinical response between baseline and week 52: frequency of use of rescue treatment | baseline and week 52 |
| Lucerne |
| Canton of Lucerne |
| Switzerland |
| Aarau Cantonal Hospital | Aarau | Switzerland |
| University Hospital Basel | Basel | 4000 | Switzerland |
| University Hospital Bern | Bern | Switzerland |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |